Benitec Biopharma
🇺🇸United States
Benitec Biopharma's BB-301 Shows Promise in OPMD Dysphagia Treatment
• Benitec Biopharma's BB-301 gene therapy demonstrates durable improvements in swallowing function in OPMD patients, according to interim clinical study data.
• Five patients have been safely treated with the low dose of BB-301 in an ongoing Phase 1b/2a clinical trial, with a sixth patient expected to be dosed soon.
• The company plans to initiate a higher dose cohort later this year, building on the positive safety and efficacy signals observed in the low-dose group.
• An interim study update will be presented at the Muscular Dystrophy Association Clinical & Scientific Conference on March 19, 2025.
Benitec Biopharma's BB-301 Gene Therapy Shows Swallowing Improvement in OPMD Patients
• Benitec Biopharma's BB-301 gene therapy demonstrated clinically meaningful improvements in swallowing for OPMD patients in a Phase 1b/2a trial.
• The first patient showed a 35% reduction in the Sydney Swallow Questionnaire (SSQ) score after 270 days of treatment.
• The second patient achieved an 89% improvement in SSQ Total Score, indicating a clinically normal swallowing profile after 180 days.
• BB-301 leverages a 'Silence and Replace' mechanism targeting the mutant PABPN1 gene, with no significant adverse events reported.
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